Research Studies Finder

The purpose of this study is to determine the safety and tolerability of an experimental form of therapy, known as WU-CART-007 ("study drug"). WU-CART-007 uses immune cells, called T cells, that are genetically changed to identify cancer cells. In this study, T cells are collected from a healthy human donor and have been modified in a laboratory. If the genetically changed T cells recognize and attach to cancer cells, they may have the ability to kill those cancer cells.

Patients with T-cell Acute Lymphoblatic Leukemia (T-ALL) or Lymphoblastic Lymphoma (LBL), where the disease has come back (relapsed) or has not responded to treatment (refractory) may be eligible for the study.

Dr. Weber at The Children's Hospital of Philadelphia is looking for boys and young men with Duchenne Muscular Dystrophy between 5-20 years old to participate in a study to learn more about bone health. Study participation includes completing two study visits: one baseline visit and one 12-month follow-up visit. To be eligible, participants must have used steroids for a minimum of 12 months by the time they complete their baseline visit. Eligible study participants will be paid for each study visit. If interested in hearing more about this study or being screened for eligibility, please contact Dr. David Weber at davidweberresearch@chop.edu.

We are trying to learn more about the ways in which adolescents (12-17 years) are involved in decision making related to their chronic condition. We are hoping to partner with families who are planning to attend or recently attended a visit in the Comprehensive Sickle Cell Center, Diabetes Center for Children, Center for Inflammatory Bowel Disease, or the Division of Rheumatology at CHOP.

If you and your child are able to help us, you will each be asked to complete a questionnaire about your experiences during a recent clinic visit. Additionally, a random selection of parents and teens will be asked to complete an interview with our team. All participants will be compensated for their participation in this study.

It is essential that we speak to families before or immediately following their scheduled visit at CHOP.

The purpose of this study is to test the safety and efficacy of the investigational drug called repotrectinib in children whose cancers have specific genetic changes (in the ALK, ROS1, or NTRK1-3 genes). This is the first study using this drug in children under 12 years of age. Repotrectinib is administered in capsule form or liquid suspension.

This clinical trial aims to test a new gene therapy in adults (18 to 35 years old) with beta thalassemia who require regular red cell transfusions. The study will evaluate the safety and efficacy of genetically engineered blood stem cells using a novel lentiviral vector that produces a high levels of a healthy beta globin. Several tests and procedures such as review of medical history, blood and urine tests, bone marrow aspirate/biopsy, heart ultrasound, electrocardiogram, magnetic resonance imaging (MRI) of the heart and liver, and lung tests will be done to find out if subjects are eligible to participate in this trial. If eligible, blood stem cells will be collected by apheresis. These stem cells will then have a healthy beta globin gene inserted. Subjects will then be hospitalized, and chemotherapy will be given to empty out the bone marrow. The stem cells, with the inserted gene (CHOP-ALS20), will then be given back to the subject. Subjects will be followed for side effects and to see if the inserted gene helps make hemoglobin and reduce or stop blood transfusions.

We are asking both parents and their child between ages 16-18 who recently received their learner's permit to partner with us as we learn more about teen driving safety. We are looking for children both with and without an ADHD diagnosis to participate. First, parents will answer questions about themselves and their child. Then, both parents and their child will complete up to five surveys over the course of two to three years. The surveys will ask about your thoughts and experiences with driving, health, and other outcomes.

The purpose of this study is to find out how well surveys can measure disease activity in kids with lower back arthritis. The study will last for either 3 or 6 months and take 2 or 3 visits. Each visit will last between 30 minutes and 2 hours and have either an MRI plus a survey or just a survey.

The purpose of this study is to find out if the study drug, MAS825, is effective in preventing flares and safe for children with NLRC4-Gain of Function (GOF). Researchers will also be testing how the body processes a drug by measuring how quickly the drug is absorbed by the body, how quickly it is broken down by the body, and how long it remains in the body (pharmacokinetics). Subjects will be asked to come into the Rheumatology clinic for blood and stool testing, physical exam, an electrocardiogram (ECG), questionnaire completion, and administration of study drug. Participation will last for up to 4 years.

This study is open to teens ages 16-20 who are about to receive or recently received their driver's license, and a parent. We are looking for teens both with and without an ADHD diagnosis. Participants must have access to a smartphone and be residents of New Jersey or Pennsylvania. We are asking if families will partner with us as we learn more about the real-world driving behaviors of teen with and without ADHD. Families will receive compensation for their participation.

The purpose of this study is to find out if the study drug, Anifrolumab, is effective and safe for pediatric participants with moderate to severe active systemic lupus erythematosus (SLE) while on background standard of care therapy. Researchers will also be testing how the body processes a drug by measuring how quickly the drug is absorbed by the body, how quickly it is broken down by the body, and how long it remains in the body (pharmacokinetics). Subjects will be asked to come into the Rheumatology clinic for blood, hepatitis/HIV, urine, and pregnancy testing, physical exam, an electrocardiogram (ECG), questionnaire completion, medical record review, corticosteroid use, and administration of study drug. Anifrolumab is administered as an infusion every 4 weeks over the course of the study.